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Trial Begins Testing Sickle Cell Blood Therapy

Red blood cells with sickle cell disease

Red blood cells with sickle cell disease (NCATS.NIH.gov)

23 June 2015. A clinical trial began enrolling participants testing a synthesized therapy for sickle cell disease to improve the flow of blood cells blocked by the condition and relieve the intense pain that results. The late-stage trial is being conducted by drug maker Pfizer Inc., with biotechnology company GlycoMimetics Inc. in Gaithersburg, Maryland.

Sickle cell disease is a genetic blood disorder affecting hemoglobin that delivers oxygen to cells in the body. People with sickle cell disease have hemoglobin molecules that cause blood cells to form into an atypical crescent or sickle shape. That abnormal shape causes the blood cells to break down, lose flexibility, and accumulate in tiny capillaries, leading to anemia and periodic painful episodes, called vaso-occlusive crises. The disease is prevalent worldwide, and affects 70,000 to 80,000 people in the U.S., including about 1 in 500 people of African descent.

In 2011, Pfizer licensed the GlycoMimetics compound code-named GMI-1070, now called Rivipansel. GlycoMimetics discovers and develops synthesized compounds that mimic the structure and activity of carbohydrates, designed to function like traditional drugs, but also to enhance biological functions more than natural carbohydrates. Rivipansel inhibits a broad range of selectins that bind carbohydrates to blood cells and vessel walls and contribute to the blockages characteristic of sickle cell disease. The drug is neither an opioid nor other general pain-reliever.

The clinical trial plans to enroll at least 350 individuals, age 6 and older, with sickle cell disease and hospitalized for a vaso-occlusive crisis. In the study, Rivipansel is being tested against a placebo, looking primarily for the time participants need to be ready for discharge from the hospital. Researchers are also measuring amount of time to actual hospital discharge, total opioid consumption, and amount of time before opioids are discontinued for participants.

Rivipansel received orphan drug designation from the U.S. Food and Drug Administration, and is being reviewed by FDA under fast-track and special protocol assessment programs. Fast-track provides an accelerated review of a new drug application, while special protocol assessment allows FDA to complete its review of a new drug before all late-stage clinical trial results are in.

Under the licensing deal with Pfizer, GlycoMimetics is responsible for early and intermediate-stage clinical trials of Rivipansel, while Pfizer conducts late-stage trials. The agreement allows, however, for a milestone payment of $20 million to GlycoMimetics upon the drug reaching late-stage trials.

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Disclosure: The author owns shares in Pfizer.

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